Gene Therapy Trial Shows Promise in Slowing Huntington's Disease

Nature reported on September 25, 2025, that Huntington's disease has been treated with gene therapy for the first time in a small clinical trial. Preliminary results from the study offer the clearest evidence to date that the progression of the inherited neurodegenerative disease can be slowed. The therapy, called AMT-130, was administered in a single dose during brain surgery and is designed to suppress the toxic protein that causes the illness.

medpath.com reported, The trial involved 29 patients, with those receiving a high dose showing a 75% reduction in disease progression over a three-year period. This development is crucial as there is currently no cure for Huntington's, a fatal disorder that causes dementia and paralysis. While the findings from the small group are notable, the results have not yet been formally peer-reviewed. Future research will need to determine if the results can be replicated in larger trials to confirm the therapy's long-term safety and efficacy.